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Gene Therapy Archives | Genetic Literacy Project

Hundreds of clinical trials are underway studying the technologys potential use in a wide range of genetic disorders, cancer and HIV/AIDS. There is some debate over whether or not the US already has approved its first gene therapy treatment.

In August 2017, the Food and Drug Administration (FDA) approved a cancer therapya CAR-T treatment marketed as Kymriahthat uses a patients own T cells and is a variation of the gene therapy that is being developed to treat single-gene diseases. The T cells are extracted and genetically altered so that they have a new gene that codes for a protein, known as a chimeric antigen receptor (CAR), that is a hybrid of two immune system proteins. One part guides the cells to the cancer cell targets and the other alerts the immune system. The cells, programmed to target and kill leukemia cells, are then injected back into the patient. Another CAR-T treatment, marketed as Yescarta, was approved for adults with aggressive forms of non-Hodgkins lymphoma in October 2017.

Some in the scientific community have pushed back against the idea of calling Kymriah or Yescarta true gene therapies, since they dont actually repair or replace a deficient gene. Instead, they say the most likely candidate to gain the first US approval is Luxturna, a one-time treatment that targets a rare, inherited form of blindness. A key committee of independent experts voted unanimously in October 2017 to recommend approval by the FDA for the treatment developed by Spark Therapeutics. The FDA is not bound by the panels decision, though the agency traditionally acts on its recommendations.

Hundreds of research studies (clinical trials) are underway to test gene therapies as treatments for genetic conditions, cancer and HIV/AIDS. ClinicalTrials.gov, a service of the National Institutes of Health, provides easy access to information about clinical trials. There is also a list of gene therapy clinical trials that are accepting (or will accept) participants. Among the studies and research: